Business mix
- The company’s products include VIMIZIM, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder.
- It also develops BRINEURA, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease.
- The company’s products under development include BMN 333, a longer-acting CNP for the treatment of multiple growth disorders, such as achondroplasia and hypochondroplasia; and BMN 351, an oligonucleotide for the treatment of duchenne muscular dystrophy.
- It serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers.
Current key questions
- Can the company's products, services, or pipeline expand adoption within biotechnology?
- How do reimbursement, regulation, clinical data, patents, and commercial execution affect growth?
- Can margins and cash flow improve while funding research, manufacturing, or go-to-market investment?
